Trial Outcomes & Findings for CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases (NCT NCT02697136)

NCT ID: NCT02697136

Last Updated: 2025-07-22

Results Overview

Change from baseline to Week 24 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

30 participants

Primary outcome timeframe

Baseline to Week 24

Results posted on

2025-07-22

Participant Flow

Participant milestones

Participant milestones
Measure	CER-001 CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions CER-001: Recombinant human apoA-I/phospholipid complexes	Placebo Saline infusion; 9 weekly infusions followed by 20 biweekly infusions Placebo: 0.9% Sodium Chloride Injection, USP
Overall Study STARTED	20	10
Overall Study Completed Week 8 (Induction Period)	18	10
Overall Study Completed Week 24 (Maintenance Period)	17	10
Overall Study COMPLETED	17	10
Overall Study NOT COMPLETED	3	0

Reasons for withdrawal

Reasons for withdrawal
Measure	CER-001 CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions CER-001: Recombinant human apoA-I/phospholipid complexes	Placebo Saline infusion; 9 weekly infusions followed by 20 biweekly infusions Placebo: 0.9% Sodium Chloride Injection, USP
Overall Study Adverse Event	3	0

Baseline Characteristics

CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CER-001 n=20 Participants CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions CER-001: Recombinant human apoA-I/phospholipid complexes	Placebo n=10 Participants Saline infusion; 9 weekly infusions followed by 20 biweekly infusions Placebo: 0.9% Sodium Chloride Injection, USP	Total n=30 Participants Total of all reporting groups
Age, Continuous	54.65 years n=5 Participants	53.27 years n=7 Participants	53.79 years n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants	6 Participants n=7 Participants	19 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	4 Participants n=7 Participants	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants	3 Participants n=7 Participants	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	15 Participants n=5 Participants	7 Participants n=7 Participants	22 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants	3 Participants n=7 Participants	11 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	12 Participants n=5 Participants	7 Participants n=7 Participants	19 Participants n=5 Participants
Region of Enrollment Canada	3 participants n=5 Participants	3 participants n=7 Participants	6 participants n=5 Participants
Region of Enrollment Netherlands	9 participants n=5 Participants	6 participants n=7 Participants	15 participants n=5 Participants
Region of Enrollment Belgium	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants	0 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Italy	2 participants n=5 Participants	0 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Israel	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment France	2 participants n=5 Participants	0 participants n=7 Participants	2 participants n=5 Participants
Mutation ApoA-I Mutation Only	8 Participants n=5 Participants	4 Participants n=7 Participants	12 Participants n=5 Participants
Mutation ABCA1 Mutation Only	11 Participants n=5 Participants	5 Participants n=7 Participants	16 Participants n=5 Participants
Mutation ApoA-I and ABCA1 Mutations	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to Week 24

Population: Modified Intent-to-Treat -- All patients randomized to treatment who have baseline and Week 24 carotid 3TMRI performed

Change from baseline to Week 24 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

Outcome measures

Outcome measures
Measure	CER-001 n=17 Participants CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions CER-001: Recombinant human apoA-I/phospholipid complexes	Placebo n=10 Participants Saline infusion; 9 weekly infusions followed by 20 biweekly infusions Placebo: 0.9% Sodium Chloride Injection, USP
Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery	-0.3 mm2 Interval -1.0 to 0.4	0.5 mm2 Interval -0.4 to 1.4

SECONDARY outcome

Timeframe: Baseline to Week 8

Population: Modified Intent-to-Treat -- All patients randomized to treatment who have baseline and Week 8 carotid 3TMRI performed

Change from baseline to Week 8 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

Outcome measures

Outcome measures
Measure	CER-001 n=16 Participants CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions CER-001: Recombinant human apoA-I/phospholipid complexes	Placebo n=10 Participants Saline infusion; 9 weekly infusions followed by 20 biweekly infusions Placebo: 0.9% Sodium Chloride Injection, USP
Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery	0.2 mm2 Interval -0.5 to 0.9	-0.5 mm2 Interval -1.4 to 0.4

SECONDARY outcome

Timeframe: Baseline to Week 48

Population: Modified Intent-to-Treat

Change from baseline to Week 48 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

Outcome measures

Outcome measures
Measure	CER-001 n=15 Participants CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions CER-001: Recombinant human apoA-I/phospholipid complexes	Placebo n=9 Participants Saline infusion; 9 weekly infusions followed by 20 biweekly infusions Placebo: 0.9% Sodium Chloride Injection, USP
Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery	-0.1 mm2 Interval -1.0 to 0.8	0.1 mm2 Interval -1.1 to 1.3

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: Carotid MRIs were analyzed for TBR in very few patients. Carotid imaging was performed for during the study, but the images were not read, measured or analyzed for TBR by the central imaging lab following termination of the study and closure of the Sponsor company (Cerenis Therapeutics). No funds were made available by the company's Board of Directors to complete these activities. At that time, paired data (baseline and 24 week) was available for only five patients.

Change from baseline to Week 24 in carotid TBR; CER-001 versus placebo; measured by FDG-PET

Outcome measures

Outcome measures
Measure	CER-001 n=4 Participants CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions CER-001: Recombinant human apoA-I/phospholipid complexes	Placebo n=1 Participants Saline infusion; 9 weekly infusions followed by 20 biweekly infusions Placebo: 0.9% Sodium Chloride Injection, USP
Change in Target to Background Ratio (TBR) of the Carotid Artery	0.30 Ratio Standard Deviation 0.160	0.35 Ratio Standard Deviation 0.215

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Weeks 8, 24 and 48

Population: Femoral imaging was performed for some patients during the study, but the images were not read, measured or analyzed for Mean Vessel Wall Area by the central imaging lab following termination of the study and closure of the Sponsor company (Cerenis Therapeutics). No funds were made available by the company's Board of Directors to complete these activities.

Assessed by 3TMRI; change from baseline; CER-001 versus placebo

Outcome measures

Outcome data not reported

Adverse Events

CER-001 DB Treatment

Serious events: 4 serious events

Other events: 19 other events

Deaths: 0 deaths

Placebo DB Treatment

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

CER-001 OL Treatment

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	CER-001 DB Treatment n=20 participants at risk CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions plus 12 biweekly infusions during the optional OL extension CER-001: Recombinant human apoA-I/phospholipid complexes	Placebo DB Treatment n=10 participants at risk Saline infusion; 9 weekly infusions followed by 20 biweekly infusions Placebo: 0.9% Sodium Chloride Injection, USP	CER-001 OL Treatment n=26 participants at risk 12 biweekly infusions during the optional OL extension CER-001: Recombinant human apoA-I/phospholipid complexes
Cardiac disorders Angina Unstable	5.0% 1/20 • Number of events 1 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).	0.00% 0/10 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).	0.00% 0/26 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).
General disorders Vascular Stent Stenosis	5.0% 1/20 • Number of events 1 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).	0.00% 0/10 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).	0.00% 0/26 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).
General disorders Complication Associated with Device	5.0% 1/20 • Number of events 1 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).	0.00% 0/10 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).	0.00% 0/26 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).
Immune system disorders Anaphylactic Reaction	5.0% 1/20 • Number of events 1 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).	0.00% 0/10 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).	0.00% 0/26 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).
Infections and infestations Pyelonephritis Acute	5.0% 1/20 • Number of events 1 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).	0.00% 0/10 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).	0.00% 0/26 • 1 year (48 weeks of double-blind treatment plus 4 weeks of post-treatment follow-up) for subjects opting out of open-label safety extension. 18 months (48 weeks of double-blind treatment followed by 26-week open-label CER-001 treatment plus 4 weeks of post-treatment follow-up) for subjects opting into open-label safety extension. Adverse events were collected by querying subjects at each study visit (weekly through Week 8, biweekly from Week 10 to 48 \[end of therapy\], at Week 52 \[4 weeks post-therapy\]). During the open-label extension, adverse events were collected biweekly from Weeks 50 through 78. Physical exams and clinical laboratory sampling to determine any adverse changes from baseline were performed at Weeks 4, 8, 16, 24, 32, 40, 48 (end of therapy) and 52 (4 weeks post-therapy).

Other adverse events

Additional Information

Constance Peyrottes

Abionyx Pharma

Phone: +33673045380

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee PI can publish results only 12 months from study completion and database lock or after the multicenter publication, whichever is sooner. All proposed publications/disclosures must be made available for sponsor review 90 days prior to public release and sponsor must respond within 45 days. The sponsor cannot require changes to the communication.
Publication restrictions are in place

Restriction type: OTHER